June 21, 2024


Cystic fibrosis is a distressing disease, clogging the lungs of children who have to fight to breathe, and shortening lives. Parents – and young adults who also suffer from it – have been on a rollercoaster ride in the last few years. They have been taken to the giddy heights of hope by the invention of drugs that for the first time work on the underlying causes of the illness where previous treatment just alleviated symptoms. And they have been plunged to the depths when those new drugs have been denied them because of their cost.

The drugs that have caused such a stir are made by the US biotech company Vertex. Its groundbreaking Kalydeco drug was licensed in Europe in 2012 and useful at first to only a small percentage of sufferers. Then came Orkambi in 2015 and Symkevi in 2018 – both two-drug combinations. Since 2020, there has been Kaftrio, which is a triple combination. Hopes rose in the UK as the combination drugs were shown to target more genetic mutations and help more people, only to be dashed by the verdict of the cost-effectiveness watchdog body, the National Institute for Health and Care Excellence (Nice). Kaftrio is the latest to be labelled unaffordable for a cash-strapped NHS.

NHS England doesn’t want to reject drugs that provide relief and even long-term improvement to children with cystic fibrosis who have no other prospects. Negotiations between NHS England and Vertex were tough, but deals have been done. In 2019, NHS England announced a two-year agreement with Vertex to buy Orkambi and Symkevi. In 2020, NHS England leapt on Kaftrio, declaring on the day that the drug was licensed that it had agreed an access package with Vertex.

But now we are on the downswing of the rollercoaster once again. As part of the deals, evidence was collected on how well cystic fibrosis patients did on the drugs as well as trial data from Vertex. Nice now says that Kaftrio, known as Trikafta in the US, is not cost-effective. Those who are on the drug should continue to get it, but new patients should not. Cue understandable distress in the cystic fibrosis community.

It would be easy to paint Nice as the villain of the piece. But it is doing the job it was set up to do. There is a good reason why it can’t green light Kaftrio. The price is astronomical. If the drug were a cure, £180,000 per patient might be justified, but it is not. It is the bill to be paid every year of that person’s life. Orkambi was suitable for about 50% of patients who had the particular genetic mutations that it targets. Kaftrio may work in more than 90% of the 10,800 people with cystic fibrosis in the UK. If the NHS chose to pay the price for these drugs, and had no extra cash, what would it have to cut? How many hip replacements? How much cancer care?

Vertex did a tremendous thing in developing drugs for a relatively rare but horrible disease that other pharma companies had ignored. But it is demanding prices that health services here and in other countries cannot pay. Vertex, predicted to make about $9bn this year from Kaftrio, says that it needs billions to invest in research. But what is the point if children are still condemned to suffer and die prematurely because nobody can afford to buy the drugs?



Source link

Leave a Reply

Your email address will not be published. Required fields are marked *